Assessing the health-related quality of life and clinical effect in children with short bowel syndrome receiving teduglutide treatment.

BACKGROUND

Short bowel syndrome (SBS) imposes a substantial burden on pediatric patients and their caregivers. Teduglutide, a glucagon-like peptide-2 (GLP-2) analog, promotes intestinal adaptation by enhancing villus hyperplasia and crypt deepening. This study aimed to evaluate its impact on the health-related quality of life (HRQoL) of children with SBS and their caregivers.

METHODS

In this matched case-control study, 24 children with SBS were enrolled (8 received teduglutide, 16 served as controls). Child HRQoL was assessed using the PedsQL™ Infant Scales and 4.0 Generic Core Module; caregiver HRQoL was evaluated with the PedsQL™ 3.0 Family Impact Module. HRQoL scores were compared between groups using independent t-tests and within the teduglutide group (pre- vs. post-treatment) using paired t-tests.

RESULTS

Baseline characteristics were comparable between groups (p > 0.05). The teduglutide group had a significantly shorter residual intestinal length (43.3 vs. 62.1 cm, P = 0.022). Treatment reduced parenteral nutrition (PN) calorie intake from 53.13 to 28.12 kcal/kg/day (P = 0.036). Teduglutide significantly improved children's total HRQoL (63.88 to 71.89, P = 0.046) and psychosocial scores (68.18 to 75.34, P = 0.027), reaching levels comparable to controls (73.53, P = 0.806; 75.84, P = 0.946, respectively). Caregivers' HRQoL also increased significantly (57.55 to 68.32, P = 0.049), surpassing that of the control group (56.80, P = 0.298).

CONCLUSIONS

Teduglutide significantly enhances HRQoL in children with SBS and their caregivers, potentially through intestinal rehabilitation and reduced PN dependence.